Gliederung

The analysis of adverse events is a key component in the assessment of a drug’s safety profile which is also required within the context of the early benefit assessment in Germany. Inappropriate analysis methods may result in misleading conclusions on a therapy’s safety and consequently benefit-risk ratio. A variety of methods are available for the analysis of adverse events, but their complexity and the imposed assumptions clearly differ. The simplest methods for contingency tables – naive proportions and derived effect measures such as relative risks and odds ratios – presuppose identical follow-up times and usually ignore recurrent events and competing risks. Consideration of varying follow-up times by means of incidence densities is possible. However, the incidence density relies on a rather restrictive constant hazard assumption. Standard procedures for event times, in turn, are based on non-informative censoring and are not readily suitable for recurrent events and competing risks. For this purpose, more complex methods in the area of event time analysis do exist; for whose application, however, the data must in turn meet the corresponding prerequisites.

The Working Group Therapeutic Research (ATF) of the German Society for Medical Informatics, Biometrics and Epidemiology (GMDS) and the Working Group Pharmaceutical Research (APF) of the German Region of the International Biometric Society (IBS-DR) have established the joint project group "Analysis of adverse events at varying follow-up times in the context of benefit assessments". Members of the project group include statisticians from academia, pharmaceutical industry and government agencies. The group has identified the research requirements for the analysis of adverse events at varying follow-up times in the benefit assessment of therapeutic interventions and is currently elaborating possible solutions. The project group will also draft and publish recommendations.

This talk will briefly outline the work of the project group, describing the six work packages on (i) standards for the collection and documentation of adverse event data, current practice in the analysis of adverse events both (ii) in Germany at IQWiG and (iii) abroad, (iv) statistical issues, (v) empirical studies, and (vi) different indications. An overview of progress on the research work, which started in September 2016, will be presented.